Successful Investigational New Drug Preparation without Reinventing the Wheel

“Black box” 101: How the Food and Drug Administration evaluates, communicates, and manages drug benefit/risk

Translating Stem Cell Studies to the Clinic for CNS Repair: Current State of the Art and the Need for a Rosetta Stone

Regulating Autologous Adult Stem Cells: The FDA Steps Up

FDA's role in anesthetic drug development.

Novel Therapies, High-Risk Pediatric Research, and the Prospect of Benefit: Learning from the Ethical Disagreements

Standardizing Safety Assessment and Reporting for Neonatal Clinical Trials

Direct-Acting Antivirals for Chronic Hepatitis C: Can Drug Properties Signal Potential for Liver Injury?

Chapter 83 - Overview of the FDA Regulatory Process

Chapter 63 - Overview of the FDA Regulatory Process

How to Design a Cell or Gene Therapy Clinical Trial: Advice from the FDA

The Food and Drug Administration (FDA) Modernization Act (the Modernization Act) of 1997 directed FDA to establish current good manufacturing practice (CGMP) requirements for positron emission tomography (PET) drugs. As directed by the US Congress in the Modernization Act, the costly CGMP regulations which the FDA applies to large pharmaceutical manufacturers are not appropriate for the PET drugs due to the unique properties of these drugs mainly the short half-lives. In consideration of the unique nature of PET drugs and PET drug production, FDA instituted specific CGMP requirements in 21 Code of Federal Regulations (CFR) part 212. Sections 1 and 2 of this book chapter illustrate the significant different aspects and the rationales for such differences between part 212 and parts 210/211 which are the CGMP requirements for non-PET drugs. The PET drug CGMP regulation found in part 212 also provides a more flexible regulatory framework for investigational PET drugs for human use produced under an investigational new drug application (IND) in accordance with part 312 and PET drugs produced with the approval of a Radioactive Drug Research Committee (RDRC) in accordance with part 361. The PET CGMP requirements for these PET drugs can be met either by compliance with part 212 or by producing such drugs in accordance with the 32nd edition of the United States Pharmacopeia (USP) General Chapter , “Radiopharmaceuticals for Positron Emission Tomography – Compounding,” which was published in 2009. In 2012, USP revised and renamed this General Chapter , “Positron Emission Tomography Drugs for Compounding, Investigational, and Research Uses.” FDA is currently considering whether to amend the PET CGMP regulations to incorporate this revised chapter into part 212. Section 4 describes format/content of the revised USP General Chapter which is very much in line with part 212 and offers more flexible requirements than the in 32nd USP.

Should women undergoing in vitro fertilization treatment or who are in the first trimester of pregnancy be vaccinated immediately against COVID-19

British chef and food activist Jamie Oliver ignited a firestorm in January 2011 when he mentioned on the Late Show with David Letterman that castoreum, a substance used to augment some strawberry and vanilla flavorings, comes from what he described as “rendered beaver anal gland.”1 The next year, vegans were outraged to learn that Starbucks used cochineal extract, a color additive derived from insect shells, to dye their strawberry Frappuccino® drinks2 (eventually, the company decided to transition to lycopene, a pigment found in tomatoes3). Although substances like castoreum and cochineal extract may be long on the “yuck factor,”4 research has shown them to be perfectly safe for most people; strident opposition arose not from safety issues but from the ingredients’ origins. But these examples demonstrate that the public often lacks significant knowledge about the ingredients in foods and where they come from. This is not a new development; the public relationship to food additives has a long history of trust lost, regained, and in some cases lost again. The Federal Food, Drug, and Cosmetic (FD&C) Act of 19385 was passed shortly after the deaths of 100 people who took an untested new form of a popular drug, which contained what turned out to be a deadly additive.6 The new law was consumer oriented and intended to ensure that people knew what was in the products they bought, and that those products were safe. The law has been amended over the years in attempts to streamline and bring order to the sprawling task of assessing and categorizing the thousands of substances used in foods, drugs, and cosmetics. One result of this streamlining is that under current U.S. law, companies can add certain types of ingredients to foods without premarket approval from the thin-stretched Food and Drug Administration (FDA). In other words, there are substances in the food supply that are unknown to the FDA. In 2010 the Government Accountability Office (GAO) concluded that a “growing number of substances … may effectively be excluded from federal oversight.”7 Is this a problem? The answer depends on whom you ask.

The Food and Drug Administration Perspective: Use of an Investigational Drug in a Medical Emergency

Old Drugs, New Concerns